dm+d

Unassigned

New Medicines

PH1, PH2 and PH3 hyperoxaluria

Information

New molecular entity
Dicerna
Dicerna

Development and Regulatory status

Phase III Clinical Trials
Phase III Clinical Trials
Phase III Clinical Trials
Yes
Yes

Category

Second-generation small interfering RNA (siRNA) therapeutic - silences the lactate dehydrogenase A (LDHA) gene, and in doing so it blocks excess production of oxalate. [2]
One estimate places the prevalence of PH type I at 1-3 cases per 1,000,000 people in the general population and the incidence at 1 case per 120,000 live births per year in Europe.[1]
PH1, PH2 and PH3 hyperoxaluria
Subcutaneous injection